[HTML][HTML] Early start of enzyme replacement therapy in pediatric male patients with classical Fabry disease is associated with attenuated disease progression
SJ Van der Veen, S Körver, A Hirsch… - Molecular Genetics and …, 2022 - Elsevier
Background Enzyme replacement therapy (ERT) slows disease progression of Fabry
disease (FD), especially when initiated before the onset of irreversible organ damage.
However, with the clinically asymptomatic progression of renal, cardiac and cerebral
disease manifestations spanning decades, optimal timing of ERT initiation remains unclear.
Methods In this cross-sectional retrospective study, seven male FD patients with a classical
disease phenotype (cFD) who started treatment with agalsidase-beta in childhood were …
disease (FD), especially when initiated before the onset of irreversible organ damage.
However, with the clinically asymptomatic progression of renal, cardiac and cerebral
disease manifestations spanning decades, optimal timing of ERT initiation remains unclear.
Methods In this cross-sectional retrospective study, seven male FD patients with a classical
disease phenotype (cFD) who started treatment with agalsidase-beta in childhood were …